Several regulatory guidances on the use of physiologically based pharmacokinetic (PBPK)
analyses and physiologically based biopharmaceutics model (s)(PBBM (s)) have been …

Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX
genes. The palliative treatment of choice is based on the use of safe and effective …

The promise of viral vector‐based gene therapy (GT) as a transformative paradigm for
treating severely debilitating and life‐threatening diseases is slowly coming to fruition with …

Given the recent success of gene therapy modalities and the growing number of cell and
gene-based therapies in clinical development across many different therapeutic areas, it is …

Recombinant adeno‐associated virus (AAV) is currently the most widely used platform for in
vivo gene therapy. Clinical pharmacology is a central field for AAV gene therapy …

The use of adeno-associated virus (AAV) as a gene delivery vehicle for secreted peptide
therapeutics can enable a new approach to durably manage chronic protein insufficiencies …

Genome editing holds the potential for curative treatments of human disease, however,
clinical realization has proven to be a challenging journey with incremental progress made …

Recombinant adeno-associated virus (rAAV) is the most effective viral vector technology for
directly translating the genomic revolution into medicinal therapies. However, the …

Neurodevelopment is a highly organized and complex process involving lasting and often
irreversible changes in the central nervous system. Inherited disorders of neurotransmission …

Introduction Unlike conventional antibodies, bispecific antibodies (bsAbs) are engineered
antibody-or antibody fragment-based molecules that can simultaneously recognize two …